2-Hydroxypropyl-β-cyclodextrin is the active component in a triple combination formulation for treatment of Niemann-Pick C1 disease

  • Jessica Davidson
  • , Elizabeth Molitor
  • , Samantha Moores
  • , Sarah E. Gale
  • , Kanagaraj Subramanian
  • , Xuntian Jiang
  • , Rohini Sidhu
  • , Pamela Kell
  • , J. Zhang
  • , Hideji Fujiwara
  • , Cristin Davidson
  • , Paul Helquist
  • , Bruce J. Melancon
  • , Michael Grigalunas
  • , Gang Liu
  • , Farbod Salahi
  • , Olaf Wiest
  • , Xin Xu
  • , Forbes D. Porter
  • , Nina H. Pipalia
  • Dana L. Cruz, Edward B. Holson, Jean E. Schaffer, Steven U. Walkley, Frederick R. Maxfield, Daniel S. Ory

Research output: Contribution to journalArticlepeer-review

21 Scopus citations

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Pharmacology, Toxicology and Pharmaceutical Science

Neuroscience

Biochemistry, Genetics and Molecular Biology